Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
CFDI STUDY with ENZYME REPLACEMENT THERAPY (ERT)
Fabry disease is a rare, inherited, genetic condition due to a deficiency of an enzyme called alpha-galactosidase A. This enzyme deficiency causes the small blood vessels to accumulate a substance called glycolipid. Without sufficient levels of the enzyme, alpha-galactosidase A, persons with Fabry Disease develop severe neuropathic pain, kidney disease, heart disease, stroke and/or premature death; often before the age of 60.
Status
Recruiting